This morning MyoKardia announced that it was being acquired by drug company Bristol Myers Squibb (BMS) for $13.1 billion. BMS plans to continue MyoKardia’s development of mavacamten as the first drug specifically intended to treat hypertrophic cardiomyopathy. The deal is expected to close before the end of 2020.
Last month, MyoKardia published positive results from its Stage 3 EXPLORER trial for obstructive HCM. Earlier this year, the FDA granted mavacamten breakthrough therapy designation which could shorten the FDA approval process by about 4 months. If all goes well, the drug could be available to HCM patients by the end of 2021.
The merger strengthens BMS’ cardiac offerings. Mavacamten will join Eliquis, a blood thinner that will go generic in 2026.
This is not MyoKardia’s first tie to Big Pharma. The French pharmaceutical company Sanofi invested $230 million into MyoKardia in 2014 but after their agreement expired in 2018, MyoKardia bought back the U.S. rights to its drugs for $80 million.
You can read a press release about the merger from MyoKardia here or a press release from BMS here. You can also read about it in the Wall Street Journal here.
DISCLOSURES: HCMBEAT HAS RECEIVED UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA. ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND IS CREDITED AS AN AUTHOR OF THE STUDY AS PUBLISHED IN THE LANCET. CYNTHIA ALSO SERVES ON MYOKARDIA’S PATIENT ADVISORY BOARD.
The results of MyoKardia’s Stage 3 EXPLORER study for its experimental drug mavacamten were presented on Saturday at the European Society of Cardiology’s virtual annual meeting and contemporaneously published in The Lancet.
The findings from the study showed that mavacamten improved exercise capacity, reduced left ventricular outflow tract gradient (responsible for the obstruction in HCM) and improved New York Heart Association functional class, correlating with a reduction in symptoms and an improvement in quality of life in patients with obstructive hypertrophic cardiomyopathy.
This was the largest placebo controlled randomized trial ever conducted in HCM. Additionally, mavacamten is the first drug specifically developed for the treatment of hypertrophic cardiomyopathy. All drugs previously used for HCM symptoms were developed to treat other conditions and were used “off label” to treat HCM.
According to Florence, Italy’s Dr. Iacopo Olivotto, who presented the results to ESC’s virtual audience, mavacamten was generally well tolerated and did not appear to have serious side effects.
MyoKardia said the Explorer-HCM data will form the basis of its submission to the FDA which is planned for the first quarter of 2021. Last month, the company announced that the drug was awarded “Breakthrough Drug” designation by the FDA which it hopes will streamline the approval process.
Also, a new trial comparing mavacamten to septal myectomy and septal alcohol ablation entitled VALOR-HCM for patients with symptomatic, obstructive HCM and who are New York Heart Association Class III – IV who meet the criteria for septal reduction therapy and have been referred for such a procedure has just gotten underway with the first patient enrolled earlier this month.
DISCLOSURES: HCMBEAT HAS RECEIVED UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA. ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL and is credited as an author of the study as published in the lancet. cynthia also serves on myokardia’s patient advisory board.
MyoKardia today announced positive topline results for its experimental drug mavacamten from its recent Stage 3 EXPLORER trial for patients with obstructive hypertrophic cardiomyopathy or HCM.
According to the press release, the drug met all primary and secondary endpoints, while relieving outflow tract obstruction, improving symptoms and enhancing quality of life for the patients enrolled in the trial. Furthermore, the drug was well tolerated and appeared to be free of significant side effects.
MyoKardia plans to seek FDA approval for mavacamten, the first drug specifically targeting the underlying biological mechanism of HCM, in the first quarter of 2021.
Full results of the EXPLORER study will be presented at a scientific meeting later this year.
DISCLOSURES: CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVES AS PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND AS A MEMBER OF MYOKARDIA’S PATIENT ADVISORY BOARD.
Two San Francisco based companies are now conducting clinical trials for three drugs specifically targeting HCM.
Continue reading “2 Companies Testing Drugs for HCM”